Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

Nathwani, Amit C, Rosales, Cecilia, McIntosh, Jenny, Rastegarlari, Ghasem, Nathwani, Devhrut, Raj, Deepak, Nawathe, Sushmita, Waddington, Simon N +16 more·2011·Molecular Therapy
DOI 10.1038/mt.2010.274

Cited by (345)

Items 101–200 of 345

  1. Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors
    Rita Ferla, Marialuisa Alliegro, Margherita Dell’Anno, Edoardo Nusco, John Michael Cullen, Stephanie N. Smith, Tyra G. Wolfsberg, Patricia O’Donnell, Ping Wang, Anh‐Dao Nguyen et al. · 2020 · Molecular Therapy — Methods & Clinical Development
  2. Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI
    Rita Ferla, Marialuisa Alliegro, Jean‐Brice Marteau, Margherita Dell’Anno, Edoardo Nusco, Séverine Pouillot, Stefania Galimberti, Maria Grazia Valsecchi, Vincent Zuliani, Alberto Auricchio · 2017 · Molecular Therapy — Methods & Clinical Development
  3. An siRNA Screen Identifies the U2 snRNP Spliceosome as a Host Restriction Factor for Recombinant Adeno-associated Viruses
    Claire A. Schreiber, Toshie Sakuma, Yoshihiro Izumiya, Sara J. Holditch, Raymond Hickey, Robert K. Bressin, Upamanyu Basu, Kazunori Koide, Aravind Asokan, Yasuhiro Ikeda · 2015 · PLoS Pathogens
  4. Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec
    Xavier M. Anguela, Katherine A. High · 2024 · Blood Advances
  5. Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema
    Ting Hu Qiu, Marı́a J. Chiuchiolo, Adele S. Whaley, Anthony R. Russo, Dolan Sondhi, Stephen M. Kaminsky, Ronald G. Crystal, Odelya E. Pagovich · 2018 · Allergy
  6. Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer
    Julien Baruteau, Dany P.B. Perocheau, Joanna Hanley, Maëlle Lorvellec, Eridan Rocha‐Ferreira, Rajvinder Karda, Joanne Ng, Natalie Suff, Juan F. Antinao Diaz, Ahad A. Rahim et al. · 2018 · Nature Communications
  7. Extracting structural and functional features of widely distributed biological circuits with single cell resolution via tissue clearing and delivery vectors
    Jennifer B. Treweek, Viviana Gradinaru · 2016 · Current Opinion in Biotechnology
  8. Role of the Vector Genome and Underlying Factor IX Mutation in Immune Responses to AAV Gene Therapy for Hemophilia B
    Rogers, Geoffrey L, Martino, Ashley T, Zolotukhin, Irene, Ertl, Hildegund CJ, Herzog, Roland W · 2014 · Journal of Translational Medicine
  9. Evaluation of Cellular Immune Response to Adeno-Associated Virus-Based Gene Therapy
    Boris Gorovits, Mitra Azadeh, George Buchlis, Michele Fiscella, Travis Harrison, Mike Havert, Sylvia Janetzki, Vibha Jawa, Brian R. Long, Yolanda D. Mahnke et al. · 2023 · The AAPS Journal
  10. Gene Therapy for Mucopolysaccharidosis Type VI Is Effective in Cats Without Pre-Existing Immunity to AAV8
    Rita Ferla, Thomas M. O’Malley, Roberto Calcedo, Patricia O’Donnell, Ping Wang, Gabriella Cotugno, Pamela Claudiani, James M. Wilson, Mark E. Haskins, Alberto Auricchio · 2012 · Human Gene Therapy
  11. Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A
    Harrison C. Brown, Philip M. Zakas, Stephan George, Ernest T. Parker, Harold Trent Spencer, Christopher B. Doering · 2018 · Molecular Therapy — Methods & Clinical Development
  12. Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity
    Mickaël Guilbaud, Marie Devaux, Celia Couzinié, Johanne Le Duff, Alice Toromanoff, Céline Vandamme, Nicolas Jaulin, Gwladys Gernoux, Thibaut Larcher, Philippe Moullier et al. · 2019 · Human Gene Therapy
  13. Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells
    Arjomandnejad, Motahareh, Sylvia, Katelyn, Blackwood, Meghan, Nixon, Thomas, Tang, Qiushi, Muhuri, Manish, Gruntman, Alisha M., Gao, Guangping, Flotte, Terence R., Keeler, Allison M. · 2021 · Molecular Therapy - Methods & Clinical Development
  14. Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy
    Fu, Haiyan, Meadows, Aaron S., Pineda, Ricardo J., Kunkler, Krista L., Truxal, Kristen V., McBride, Kim L., Flanigan, Kevin M., McCarty, Douglas M. · 2017 · Human Gene Therapy Clinical Development
  15. Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia
    Glenn F. Pierce, Sylvia Fong, Brian R. Long, Radosław Kaczmarek · 2023 · Journal of Thrombosis and Haemostasis
  16. Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans
    Gwladys Gernoux, Alisha M. Gruntman, Meghan Blackwood, Marina V. Zieger, Terence R. Flotte, Christian Mueller · 2020 · Molecular Therapy
  17. Effective depletion of pre-existing anti-AAV antibodies requires broad immune targeting
    Velazquez, Victoria M., Meadows, Aaron S., Pineda, Ricardo J., Camboni, Marybeth, McCarty, Douglas M., Fu, Haiyan · 2017 · Molecular Therapy - Methods & Clinical Development
  18. Coagulation Factor IX for Hemophilia B Therapy
    Nadezhda A. Orlova, Sergey V. Kovnir, Ivan Ivanovich Vorobiev, А. Г. Габибов · 2012 · Acta Naturae
  19. Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques
    Nicola Brunetti‐Pierri, Aimée Liou, Priti Patel, Donna J Palmer, Nathan C. Grove, Milton J. Finegold, Pasquale Piccolo, Elizabeth Donnachie, Karen Rice, Arthur L. Beaudet et al. · 2012 · Molecular Therapy
  20. Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies
    Timothy C. Nichols, Christine A. Hough, Henrik Agersø, Mirella Ezban, David Lillicrap · 2016 · Journal of Thrombosis and Haemostasis
  21. Gene Therapy for Hemophilia
    Amit Chunilal Nathwani, Andrew M. Davidoff, Edward G. D. Tuddenham · 2017 · Hematology/Oncology Clinics of North America
  22. Riboswitches for Controlled Expression of Therapeutic Transgenes Delivered by Adeno-Associated Viral Vectors
    Zachary J. Tickner, Michael Farzan · 2021 · Pharmaceuticals
  23. Current advances in gene therapy of mitochondrial diseases
    Vladislav O. Soldatov, Marina V. Kubekina, M. Yu. Skorkina, А. Е. Белых, Tatiana V. Egorova, Михаил Викторович Корокин, Mikhail V. Pokrovskiy, Alexey V. Deykin, Plamena R. Angelova · 2022 · Journal of Translational Medicine
  24. Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates
    Douglas Ballon, Jonathan B. Rosenberg, Edward K. Fung, Anastasia Nikolopoulou, Paresh J. Kothari, Bishnu P. De, Bin He, Alvin Chen, Linda Heier, Dolan Sondhi et al. · 2020 · Human Gene Therapy
  25. Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
    Coy Don Heldermon, Elizabeth Y. Qin, Kevin K. Ohlemiller, Erik D. Herzog, Jeremy R. Glissen Brown, Carole A. Vogler, Wei Hou, John L. Orrock, Brett E. Crawford, Mark S. Sands · 2013 · Gene Therapy
  26. Sustained expression and safety of human GNE in normal mice after gene transfer based on AAV8 systemic delivery
    Stella Mitrani‐Rosenbaum, Lena Yakovlev, Michal Becker‐Cohen, Michal Telem, Moran Elbaz, Nurit Yanay, Hagit Yotvat, Uri Ben Shlomo, Avi Harazi, Yakov Fellig et al. · 2012 · Neuromuscular Disorders
  27. Gene therapy for metabolic diseases
    R. Chandler, C. Venditti · 2016 · Translational Science of Rare Diseases
  28. CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome
    Wu, Te-Lang, Li, Hua, Faust, Susan M, Chi, Emily, Zhou, Shangzhen, Wright, Fraser, High, Katherine A, Ertl, Hildegund CJ · 2014 · Molecular Therapy
  29. AAV-Mediated Gene Delivery to the Liver: Overview of Current Technologies and Methods
    Brett Palaschak, Roland W. Herzog, David M. Markusic · 2019 · Methods in molecular biology
  30. Progress and challenges in development of new therapies for urea cycle disorders
    Leandro R. Soria, Nicholas Ah Mew, Nicola Brunetti‐Pierri · 2019 · Human Molecular Genetics
  31. AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects
    Katarzyna Chwalenia, Vivi-Yun Feng, Nicole Hemmer, Hans J. Friedrichsen, Ioulia Vorobieva, Matthew J. A. Wood, Thomas C. Roberts · 2025 · Gene Therapy
  32. Limitations of Encapsidation of Recombinant Self-Complementary Adeno-Associated Viral Genomes in Different Serotype Capsids and Their Quantitation
    Yuan Wang, Chen Ling, Liujiang Song, Lina Wang, George Aslanidi, Mengqun Tan, Changquan Ling, Arun Srivastava · 2012 · Human Gene Therapy Methods
  33. Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality
    Marti Cabanes‐Creus, Samantha L. Ginn, Anais K. Amaya, Sophia H.Y. Liao, Adrian Westhaus, Claus V. Hallwirth, Patrick Wilmott, Jason Ward, Kimberley L. Dilworth, Giorgia Santilli et al. · 2018 · Molecular Therapy — Methods & Clinical Development
  34. Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
    Julien Baruteau, Sharon C. Cunningham, Berna Şeker Yılmaz, Dany P.B. Perocheau, Simon Eaglestone, Derek G. Burke, Adrian James Thrasher, Simon N. Waddington, Leszek Lisowski, Ian E. Alexander et al. · 2021 · Molecular Therapy — Methods & Clinical Development
  35. Gene therapy for hemophilia – From basic science to first approvals of “one-and-done” therapies
    Roland W. Herzog, Radosław Kaczmarek, Katherine A. High · 2025 · Molecular Therapy
  36. Cardiovascular RNA Interference Therapy
    Wolfgang C. Poller, Juliane Tank, Carsten Skurk, Martina Gast · 2013 · Circulation Research
  37. Haemophilia gene therapy: Progress and challenges
    Elsa Lhériteau, Andrew M. Davidoff, Amit Chunilal Nathwani · 2015 · Blood Reviews
  38. Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
    Sara Marcó, Virginia Haurigot, Maria Luisa Jaén, Albert Ribera, Víctor Sánchez, Maria Molas, Miguel Garcia, Xavier León, Carles Roca, Xavier Sánchez et al. · 2021 · Molecular Therapy — Methods & Clinical Development
  39. Preferential Targeting of Disseminated Liver Tumors Using a Recombinant Adeno-Associated Viral Vector
    Marco Della Peruta, Adam Badar, Cecilia Rosales, Shilpa Chokshi, Azadeh Kia, Devhrut Nathwani, Eva Galante, Ran Yan, Erik Årstad, Andrew M. Davidoff et al. · 2015 · Human Gene Therapy
  40. The Unusual Properties of the AAV Inverted Terminal Repeat
    Kenneth I. Berns · 2020 · Human Gene Therapy
  41. Modeling Pulmonary Disease Pathways Using Recombinant Adeno-Associated Virus 6.2
    Benjamin Strobel, Matthias J. Duechs, Ramona Schmid, Birgit Stierstorfer, Hannes Bucher, Karsten Quast, Detlef Stiller, Tobias Hildebrandt, Detlev Mennerich, Florian Gantner et al. · 2015 · American Journal of Respiratory Cell and Molecular Biology
  42. Preclinical Dose-Finding Study With a Liver-Tropic, Recombinant AAV-2/8 Vector in the Mouse Model of Galactosialidosis
    Hu, Huimin, Gomero, Elida, Bonten, Erik, Gray, John T, Allay, Jim, Wu, Yanan, Wu, Jianrong, Calabrese, Christopher, Nienhuis, Arthur, d'Azzo, Alessandra · 2012 · Molecular Therapy
  43. Molecular Engineering of Adeno-Associated Virus Capsid Improves Its Therapeutic Gene Transfer in Murine Models of Hemophilia and Retinal Degeneration
    Bertin Mary, Shubham Maurya, Mohit Kumar, Sridhar Bammidi, Vikas S. Kumar, Giridhara Rao Jayandharan · 2019 · Molecular Pharmaceutics
  44. Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia
    Elizabeth A. Harrington, Jennifer L. Sloan, Irini Manoli, Randy J. Chandler, Mark Schneider, Peter J. McGuire, Roberto Calcedo, James M. Wilson, Charles P. Venditti · 2016 · Human Gene Therapy
  45. Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs
    Elisabeth A. Spronck, Ying Poi Liu, Jacek Lubelski, Erich Ehlert, Sander Gielen, Paula S. Montenegro-Miranda, Martin de Haan, Bart A. Nijmeijer, Valérie Ferreira, Harald Petry et al. · 2019 · Molecular Therapy — Methods & Clinical Development
  46. Therapeutic expression of human clotting factors IX and × following adeno‐associated viral vector‐mediated intrauterine gene transfer in early‐gestation fetal macaques
    Jerry Kok Yen Chan, Irene Gil-Fariña, Nuryanti Binti Johana, Cecilia Rosales, Yi Wan Tan, Jessika Ceiler, Jenny McIntosh, Bryan Emmett Ogden, Simon N. Waddington, Manfred Schmidt et al. · 2018 · The FASEB Journal
  47. Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research
    Stefania Nicola, Giovanni Rolla, Luisa Brussino · 2019 · Drugs in Context
  48. Adeno‐associated virus 8‐mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models
    Aaron D. Black, Vidyullatha Vasireddy, D. Chung, A. Maguire, R. Gaddameedi, T. Tolmachova, M. Seabra, J. Bennett · 2014 · Journal of Gene Medicine
  49. Prevalence of Anti–Adeno-Associated Virus Serotype 8 Neutralizing Antibodies and Arylsulfatase B Cross-Reactive Immunologic Material in Mucopolysaccharidosis VI Patient Candidates for a Gene Therapy Trial
    Rita Ferla, Pamela Claudiani, Marco Savarese, Karen Kozarsky, Rossella Parini, Maurizio Scarpa, Maria Alice Donati, Giovanni Sorge, John J. Hopwood, Giancarlo Parenti et al. · 2015 · Human Gene Therapy
  50. Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation
    Aaron S. Meadows, Ricardo J. Pineda, Laurie Goodchild, Tierra A. Bobo, Haiyan Fu · 2019 · Molecular Therapy — Methods & Clinical Development
  51. TLR9-independent CD8+ T cell responses in hepatic AAV gene transfer through IL-1R1-MyD88 signaling
    Kumar, Sandeep R.P., Biswas, Moanaro, Cao, Di, Arisa, Sreevani, Muñoz-Melero, Maite, Lam, Anh K., Piñeros, Annie R., Kapur, Reuben, Kaisho, Tsuneyasu, Kaufman, Randal J. et al. · 2024 · Molecular Therapy
  52. Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis
    C. Aalbers, L. Bevaart, S. Loiler, Karin de Cortie, J. F. Wright, F. Mingozzi, P. Tak, M. Vervoordeldonk · 2015 · PLoS ONE
  53. Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease
    Rita Ferla, Pamela Claudiani, Gabriella Cotugno, Paola Saccone, Elvira De Leonibus, Alberto Auricchio · 2014 · Human Gene Therapy
  54. Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia
    David M. Markusic, Roland W. Herzog · 2013 · Journal of Genetic Syndromes & Gene Therapy
  55. Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep
    Marcus G. Davey, John Samuel Riley, Abigail Andrews, Alec Tyminski, Maria P. Limberis, Jennifer E. Pogoriler, Emily Anne Partridge, Aliza M. Olive, Holly L. Hedrick, Alan W. Flake et al. · 2017 · PLoS ONE
  56. Gene therapy for inherited metabolic diseases
    Berna Şeker Yılmaz, Sonam Gurung, Dany P.B. Perocheau, John Robert Counsell, Julien Baruteau · 2020 · PubMed Central
  57. The Perspective of DMPK on Recombinant Adeno-Associated Virus-Based Gene Therapy: Past Learning, Current Support, and Future Contribution
    Nancy Chen, Kefeng Sun, Nagendra Venkata Chemuturi, Hyelim Cho, Cindy Q. Xia · 2022 · The AAPS Journal
  58. Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates
    Samantha Powers, Shibi B. Likhite, Kamal K.E. Gadalla, Carlos J. Miranda, Amy J. Huffenberger, Cassandra N. Dennys, Kevin D. Foust, Pablo R. Morales, Christopher R. Pierson, Federica Rinaldi et al. · 2023 · Molecular Therapy
  59. Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation
    David William Scott, Jay Nelson Lozier · 2011 · British Journal of Haematology
  60. Advancements in adeno-associated viral gene therapy approaches: exploring a new horizon
    Caroline J. Aalbers, Paul Peter Tak, Margriet J. Vervoordeldonk · 2011 · F1000 Medicine Reports
  61. Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs
    Mary Beth Callan, Mark E. Haskins, Ping Wang, Shangzhen Zhou, Katherine A. High, Valder R. Arruda · 2016 · PLoS ONE
  62. Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism
    Virginia Maria Ginocchio, Rita Ferla, Alberto Auricchio, Nicola Brunetti‐Pierri · 2019 · Human Gene Therapy
  63. Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy
    Raquel Cabrera-Pérez, Ferran Vila‐Julià, Michio Hirano, Federico Mingozzi, Javier Torres‐Torronteras, Ramón Martí · 2019 · Human Gene Therapy
  64. Gene Therapy Approaches for the Treatment of Hemophilia B
    Anastasiia B. Soroka, Sofya G. Feoktistova, Olga N. Mityaeva, Pavel Volchkov · 2023 · International Journal of Molecular Sciences
  65. Advances in understanding disease mechanisms and potential treatments for Crigler–Najjar syndrome
    Giulia Bortolussi, Andrés Fernando Muro · 2018 · Expert Opinion on Orphan Drugs
  66. AAV5-miHTT-mediated huntingtin lowering improves brain health in a Huntington’s disease mouse model
    Sarah B. Thomson, Anouk Stam, Cynthia C. Brouwers, Valentina Fodale, Alberto Bresciani, Michael Vermeulen, Sara Mostafavi, Terri L. Petkau, Austin Hill, Andrew C. Yung et al. · 2022 · Brain
  67. Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges
    Deepak Raj, Andrew M. Davidoff, Amit Chunilal Nathwani · 2011 · Expert Review of Hematology
  68. Clinical and Translational Landscape of Viral Gene Therapies
    Alexandra Yudaeva, Anastasiya Kostyusheva, Artyom Kachanov, Sergey Brezgin, Natalia Ponomareva, Alessandro Parodi, Vadim S. Pokrovsky, Alexander N. Lukashev, Vladimir Chulanov, Dmitry S. Kostyushev · 2024 · Cells
  69. Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodents
    Ina Rittelmeyer, Michael Rothe, Martijn H. Brugman, Marcus Iken, Axel Schambach, Michael Peter Manns, Christopher Baum, Ute Modlich, Michael Ott · 2012 · Hepatology
  70. A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene
    Sem J. Aronson, Robert S. Bakker, Sascha Moenis, Remco van Dijk, Giulia Bortolussi, Fanny Collaud, Xiaoxia Shi, Suzanne Duijst, Lysbeth ten Bloemendaal, Giuseppe Ronzitti et al. · 2020 · Molecular Therapy — Methods & Clinical Development
  71. Intravenous AAV8 Encoding Urocortin-2 Increases Function of the Failing Heart in Mice
    N. Chin Lai, Mei Hua Gao, Dimosthenis Giamouridis, Jorge Suárez, Atsushi Miyanohara, Jay Parikh, Stephen Hightower, Tracy Guo, WOLFGANG H. DILLMANN, Young Chul Kim et al. · 2015 · Human Gene Therapy
  72. Proteasome Inhibition Is Partially Effective in Attenuating Pre-Existing Immunity against Recombinant Adeno-Associated Viral Vectors
    József Kármán, Nathan Gumlaw, Jinhua Zhang, Ji-Lei Jiang, Seng H. Cheng, Yunxiang Zhu · 2012 · PLoS ONE
  73. Effects of FVIII immunity on hepatocyte and hematopoietic stem cell–directed gene therapy of murine hemophilia A
    Allison M. Lytle, Harrison C. Brown, Na Yoon Paik, Kristopher A. Knight, John Fraser Wright, Harold Trent Spencer, Christopher B. Doering · 2016 · Molecular Therapy — Methods & Clinical Development
  74. Ultramicroscopy as a novel tool to unravel the tropism of AAV gene therapy vectors in the brain
    Sandro Alves, Julia Bode, Alexis‐Pierre Bemelmans, Christof V. Kalle, Nathalie Cartier, Björn Tews · 2016 · Scientific Reports
  75. Homologous Recombination-Based Genome Editing by Clade F AAVs Is Inefficient in the Absence of a Targeted DNA Break
    Geoffrey L. Rogers, Hsu-Yu Chen, Heidy Morales, Paula M. Cannon · 2019 · Molecular Therapy
  76. Marginal integrity of self- and total-etching adhesives in two different application protocols.
    Claus-Peter Ernsta, Tim Kötter, Anja Victor, Kerem Canbek, Mathias Brandenbusch, Brita Willershausen · 2004 · PubMed
  77. Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice
    Michela Lisjak, Alessia De Caneva, Thibaut Marais, Elena Barbon, Maria Grazia Biferi, Fabiola Porro, Adi Barzel, Lorena Zentilin, Mark A. Kay, Federico Mingozzi et al. · 2022 · Frontiers in Genome Editing
  78. Enhanced T Cell Function in a Mouse Model of Human Glycosylation
    George Buchlis, Pamela M. Odorizzi, Paula C. Soto, Oliver M.T. Pearce, Daniel J. Hui, Martha S. Jordan, Ajit P Varki, E. John Wherry, Katherine A. High · 2013 · The Journal of Immunology
  79. Optimization of Dexamethasone Administration for Maintaining Global Transduction Efficacy of Adeno-Associated Virus Serotype 9
    Zheng Chai, Xintao Zhang, Amanda Lee Dobbins, Kelly Michelle Rigsbee, Bing Wang, Richard Jude Samulski, Chengwen Li · 2019 · Human Gene Therapy
  80. Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
    Shao, Wenwei, Pei, Xiaolei, Cui, Caibin, Askew, Charles, Dobbins, Amanda, Chen, Xiaojing, Abajas, Yasmina L., Gerber, David A., Samulski, R. Jude, Nichols, Timothy C. et al. · 2019 · Gene Therapy
  81. Adeno‐Associated Virus Vector–Based Gene Therapy for Monogenetic Metabolic Diseases of the Liver
    Norman Junge, Federico Mingozzi, Michael Ott, Ulrich Baumann · 2015 · Journal of Pediatric Gastroenterology and Nutrition
  82. Platelet and endothelial expression of clotting factors for the treatment of hemophilia
    Robert R. Montgomery, Qizhen Shi · 2012 · Thrombosis Research
  83. Bioanalysis of Adeno-Associated Virus Gene Therapy Therapeutics: Regulatory Expectations
    Boris Gorovits, Jean-Claude A. Marshall, Justin Smith, Laurence O. Whiteley, Hendrik Neubert · 2019 · Bioanalysis
  84. Fetal gene therapy: recent advances and current challenges
    Citra Nurfarah Zaini Mattar, Mahesh Arjandas Choolani, Arijit Biswas, Simon N. Waddington, Jerry Kok Yen Chan · 2011 · Expert Opinion on Biological Therapy
  85. Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell
    Sneha Sitaraman, Konstantinos–Dionysios Alysandratos, Jennifer Wambach, Maria P. Limberis · 2022 · Human Gene Therapy
  86. Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
    Britt Hanson, Sofia Stenler, Nina Ahlskog, Katarzyna Chwalenia, Nenad Svrzikapa, Anna M. L. Coenen-Stass, Marc S. Weinberg, Matthew J. A. Wood, Thomas C. Roberts · 2022 · Molecular Therapy — Nucleic Acids
  87. Gene therapies for mucopolysaccharidoses
    Alessandro Rossi, Nicola Brunetti‐Pierri · 2023 · Journal of Inherited Metabolic Disease
  88. New and improved AAVenues: current status of hemophilia B gene therapy
    Mark A. Brimble, Ulrike M. Reiss, Amit Chunilal Nathwani, Andrew M. Davidoff · 2015 · Expert Opinion on Biological Therapy
  89. Gene therapy for haemophilia B
    Edward G. D. Tuddenham · 2012 · Haemophilia
  90. Retrograde capabilities of adeno-associated virus vectors in the central nervous system
    Magdalena Surdyka, Maciej Figiel · 2021 · Biotechnologia
  91. Restoring Ciliary Function: Gene Therapeutics for Primary Ciliary Dyskinesia
    Nicholas W. Keiser, E. Cant, Sneha Sitaraman, A. Shoemark, M. Limberis · 2023 · Human Gene Therapy
  92. Perinatal Gene Transfer to the Liver
    Tristan R. McKay, Ahad A. Rahim, Suzanne M. K. Buckley, Natalie J Ward, Jerry Kok Yen Chan, Steven J. Howe, Simon N. Waddington · 2011 · Current Pharmaceutical Design
  93. An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8+ T Cells
    Palaschak, Brett, Marsic, Damien, Herzog, Roland W., Zolotukhin, Sergei, Markusic, David M. · 2017 · Molecular Therapy - Methods & Clinical Development
  94. Inhibitors – cellular aspects and novel approaches for tolerance
    David William Scott · 2014 · Haemophilia
  95. Bile-duct proliferation as an unexpected side-effect after AAV2-LDLR gene transfer to rabbit liver
    Elisa Hytönen, Anniina Laurema, Hanna M Kankkonen, Atsushi Miyanohara, Vesa J. Kärjä, Mika Hujo, Nihay Laham-Karam, Seppo Ylä‐Herttuala · 2019 · Scientific Reports
  96. Biodistribution and Shedding of AAV Vectors
    Caroline Le Guiner, Phillipe Moullier, Valder R. Arruda · 2011 · Methods in molecular biology
  97. Dexamethasone Transiently Enhances Transgene Expression in the Liver When Administered at Late-Phase Post Long-Term Adeno-Associated Virus Transduction
    Zheng Chai, Xintao Zhang, Amanda Lee Dobbins, Richard Jude Samulski, Elizabeth P. Merricks, Timothy C. Nichols, Chengwen Li · 2021 · Human Gene Therapy
  98. Motor Neuron‐Specific Membrane Depolarization of Transected Peripheral Nerves by Upconversion Nanoparticle‐Mediated Optogenetics
    Jun Yan, Yunjing Wan, Zhe Ji, Chenxi Li, Chengcheng Tao, Yunjiao Tang, Yuze Zhang, Yaobo Liu, Jian Liu · 2023 · Advanced Functional Materials
  99. Development of a liver‐specific Tet‐off AAV8 vector for improved safety of insulin gene therapy for diabetes
    Shu Uin Gan, Zhenying Fu, Kian Chuan Sia, Oi Lian Kon, Roy Calne, Kok‐Onn Lee · 2018 · The Journal of Gene Medicine
  100. The case for intrauterine gene therapy
    Citra Nurfarah Zaini Mattar, Simon N. Waddington, Arijit Biswas, Andrew M. Davidoff, Mahesh Arjandas Choolani, Jerry Kok Yen Chan, Amit Chunilal Nathwani · 2012 · Best Practice & Research Clinical Obstetrics & Gynaecology